CYP46A1 gene for the treatment of alzheimer's disease
AssigneeInstitut National De La Sante Et De La Recherche Medicale (Inserm)
InventorsPatrick Aubourg, Nathalie Cartier-Lacave, Eloise Hudry
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Abstract
The invention relates to a viral vector for treating Alzheimers disease, which vector comprises a cholesterol 24-hydroxylase (CYP46A1) encoding nucleic acid. In a preferred embodiment, the viral vector may be an Adeno-Associated-Virus (AAV) vector, preferably an AVV5 vector. The vector may be useful for the manufacture of a pharmaceutical composition for the treatment of Alzheimers disease in a subject, wherein the vector is to be administered directly into the brain of the subject or by intravenous or intrathecal injection.
Claims13
A method for treating a subject having Alzheimer's disease, said method comprising the steps of:
- (a) providing a viral vector comprising a cholesterol 24-hydroxylase encoding nucleic acid;
- and
- (b) delivering the viral vector to the central nervous system (CNS) of the subject, whereby said vector transduces cells in the CNS, and whereby cholesterol 24-hydroxylase is expressed by the transduced cells at a therapeutically effective level, wherein expression of the cholesterol 24-hydroxylase gene results in a reduction in amyloid plaques in the treated subject having Alzheimer's disease as compared to a non-treated subject having Alzheimer's disease.
Dependencies are inferred from the claim text.